JP2013518899A5 - - Google Patents
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- JP2013518899A5 JP2013518899A5 JP2012552100A JP2012552100A JP2013518899A5 JP 2013518899 A5 JP2013518899 A5 JP 2013518899A5 JP 2012552100 A JP2012552100 A JP 2012552100A JP 2012552100 A JP2012552100 A JP 2012552100A JP 2013518899 A5 JP2013518899 A5 JP 2013518899A5
- Authority
- JP
- Japan
- Prior art keywords
- genome
- aav
- vector
- dystrophin
- composition according
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- GXJABQQUPOEUTA-RDJZCZTQSA-N Bortezomib Chemical compound C([C@@H](C(=O)N[C@@H](CC(C)C)B(O)O)NC(=O)C=1N=CC=NC=1)C1=CC=CC=C1 GXJABQQUPOEUTA-RDJZCZTQSA-N 0.000 claims 12
- 229960001467 bortezomib Drugs 0.000 claims 12
- 239000008194 pharmaceutical composition Substances 0.000 claims 12
- 102000001690 Factor VIII Human genes 0.000 claims 10
- 108010054218 Factor VIII Proteins 0.000 claims 10
- 229960000301 Factor VIII Drugs 0.000 claims 10
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 8
- 229940099039 Velcade Drugs 0.000 claims 6
- 239000000203 mixture Substances 0.000 claims 6
- 102000001039 Dystrophin Human genes 0.000 claims 5
- 108010069091 Dystrophin Proteins 0.000 claims 5
- 108020004707 nucleic acids Proteins 0.000 claims 5
- 150000007523 nucleic acids Chemical class 0.000 claims 5
- 102000034448 gene-regulatory proteins Human genes 0.000 claims 4
- 108091006088 gene-regulatory proteins Proteins 0.000 claims 4
- 241000432074 Adeno-associated virus Species 0.000 claims 3
- 229920001405 Coding region Polymers 0.000 claims 3
- 210000004027 cells Anatomy 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 239000003937 drug carrier Substances 0.000 claims 2
- 239000000825 pharmaceutical preparation Substances 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 102100000368 F8 Human genes 0.000 claims 1
- 101700070229 F8 Proteins 0.000 claims 1
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 210000004185 Liver Anatomy 0.000 claims 1
- 208000003627 Muscular Dystrophy Diseases 0.000 claims 1
- 201000003883 cystic fibrosis Diseases 0.000 claims 1
- 201000003542 factor VIII deficiency Diseases 0.000 claims 1
- 210000000663 muscle cells Anatomy 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
Claims (19)
（ｂ）ボルテゾミブ（ベルケイド（登録商標））と
を含む組成物。 (A) an adeno-associated virus (AAV) vector comprising a heterologous nucleic acid encoding a protein selected from the group consisting of Factor VIII (FVIII), dystrophin, mini-dystrophin and transmembrane regulator protein (CFTR) , A vector in which the genome of the AAV vector is excessive compared to the wild type AAV genome;
(B) A composition comprising bortezomib (Velcade (registered trademark)) .
（ａ）前記核酸を含むアデノ随伴ウイルス（ＡＡＶ）ベクターであって、ＡＡＶベクターのゲノムが、野生型ＡＡＶゲノムに比して過大であるベクターと、
（ｂ）ボルテゾミブ（ベルケイド（登録商標））と
を薬学的に許容される担体中に含む医薬製剤。 A pharmaceutical formulation for delivering to a cell a nucleic acid encoding a protein selected from the group consisting of Factor VIII (FVIII), dystrophin, mini-dystrophin and transmembrane regulator protein (CFTR) ,
(A) an adeno-associated virus (AAV) vector comprising the nucleic acid, wherein the AAV vector genome is larger than the wild-type AAV genome;
(B) Bortezomib (Velcade (registered trademark)) and
Including pharmaceutical preparations in a pharmaceutically acceptable carrier a.
（ｂ）ボルテゾミブ（ベルケイド（登録商標））と
を含むキット。 (A) an adeno-associated virus (AAV) vector comprising a heterologous nucleic acid encoding a protein selected from the group consisting of Factor VIII (FVIII), dystrophin, mini-dystrophin and transmembrane regulator protein (CFTR) , A vector in which the genome of the AAV vector is excessive compared to the wild type AAV genome;
(B) A kit containing bortezomib (Velcade (registered trademark)) .
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US30199810P | 2010-02-05 | 2010-02-05 | |
| US61/301,998 | 2010-02-05 | ||
| PCT/US2011/023715 WO2011097456A2 (en) | 2010-02-05 | 2011-02-04 | Compositions and methods for enhanced parvovirus transduction |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013518899A JP2013518899A (en) | 2013-05-23 |
| JP2013518899A5 true JP2013518899A5 (en) | 2015-02-26 |
| JP6141021B2 JP6141021B2 (en) | 2017-06-07 |
Family
ID=44356082
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012552100A Active JP6141021B2 (en) | 2010-02-05 | 2011-02-04 | Compositions and methods for enhancing parvovirus transduction |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US9169492B2 (en) |
| EP (1) | EP2531604B1 (en) |
| JP (1) | JP6141021B2 (en) |
| CA (1) | CA2788682C (en) |
| ES (1) | ES2628889T3 (en) |
| WO (1) | WO2011097456A2 (en) |
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| CA2909085C (en) * | 2013-04-08 | 2023-08-29 | University Of Iowa Research Foundation | Chimeric adeno-associated virus/ bocavirus parvovirus vector |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
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| KR20230145206A (en) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | Modulatory polynucleotides |
| EP3230441A4 (en) | 2014-12-12 | 2018-10-03 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| EP3978614A3 (en) * | 2015-01-07 | 2022-07-27 | Universitat Autònoma de Barcelona | Single-vector gene construct comprising insulin and glucokinase genes |
| EP3611186A1 (en) * | 2015-02-06 | 2020-02-19 | The University of North Carolina at Chapel Hill | Optimized human clotting factor viii gene expression cassettes and their use |
| CN108697813B (en) * | 2016-01-13 | 2024-01-16 | 勃林格殷格翰动物保健美国公司 | Recombinant AAV vectors expressing a bone protective gene comprising HAS2 and lubricin for use in the treatment of osteoarthritis and related joint disorders in a mammal |
| US11702672B2 (en) | 2016-02-08 | 2023-07-18 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| CA3016985C (en) | 2016-03-07 | 2023-07-04 | University Of Iowa Research Foundation | Aav-mediated expression using a synthetic promoter and enhancer |
| EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| CA3022634A1 (en) * | 2016-05-03 | 2017-11-09 | Wayne State University | Method of enhancing viral-mediated gene delivery in the eye using proteosome inhibitors |
| RU2764587C2 (en) | 2016-05-18 | 2022-01-18 | Вояджер Терапьютикс, Инк. | Methods and compositions for treatment of huntington's chorea |
| MX2018014154A (en) | 2016-05-18 | 2019-05-06 | Voyager Therapeutics Inc | Modulatory polynucleotides. |
| EP3500696A4 (en) | 2016-08-16 | 2020-04-08 | Bluebird Bio, Inc. | Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use |
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| EP3831281A1 (en) | 2016-08-30 | 2021-06-09 | The Regents of The University of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| CN109923211A (en) | 2016-09-08 | 2019-06-21 | 蓝鸟生物公司 | PD-1 homing endonuclease variants, composition and application method |
| JP7060591B2 (en) | 2016-10-17 | 2022-04-26 | ２セブンティ バイオ インコーポレイテッド | TGFβR2 endonuclease variant, composition, and method of use |
| KR102653324B1 (en) | 2016-11-17 | 2024-04-01 | 2세븐티 바이오, 인코포레이티드 | TGFβ signal converter |
| US11142775B2 (en) | 2017-01-13 | 2021-10-12 | University Of Iowa Research Foundation | Bocaparvovirus small noncoding RNA and uses thereof |
| US10436089B2 (en) * | 2017-01-18 | 2019-10-08 | Cummins Emission Solutions Inc. | Radio frequency sensor in an exhaust aftertreatment system |
| AU2018221730B2 (en) | 2017-02-15 | 2024-06-20 | 2Seventy Bio, Inc. | Donor repair templates multiplex genome editing |
| EP3619308A4 (en) | 2017-05-05 | 2021-01-27 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| AU2018261790A1 (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| CA3064014A1 (en) | 2017-05-25 | 2018-11-29 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
| JOP20190269A1 (en) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| WO2019018342A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| WO2019028306A2 (en) | 2017-08-03 | 2019-02-07 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| US11779654B2 (en) | 2017-10-04 | 2023-10-10 | 2Seventy Bio, Inc. | PCSK9 endonuclease variants, compositions, and methods of use |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| EP4124658A3 (en) | 2017-10-16 | 2023-04-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| US20210154328A1 (en) * | 2018-04-11 | 2021-05-27 | Cancer Targeting System, Inc. | Therapeutic constructs for treating cancer |
| US20210261659A1 (en) | 2018-06-14 | 2021-08-26 | Bluebird Bio, Inc. | Cd79a chimeric antigen receptors |
| WO2020072059A1 (en) | 2018-10-04 | 2020-04-09 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
| US20220195461A1 (en) * | 2019-04-15 | 2022-06-23 | University Of Iowa Research Foundation | Methods and compositions for transgene expression |
| US20220347298A1 (en) * | 2019-10-04 | 2022-11-03 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant aav |
| BR112022020444A2 (en) | 2020-04-10 | 2022-11-29 | Sola Biosciences Llc | COMPOSITIONS AND METHODS FOR THE TREATMENT OF PROTEIN AGGREGATION DISORDERS |
| WO2021222168A2 (en) | 2020-04-28 | 2021-11-04 | Sola Biosciences Llc | Compositions and methods for the treatment of tdp-43 proteinopathies |
| WO2021248038A1 (en) | 2020-06-05 | 2021-12-09 | Sola Biosciences Llc | Compositions and methods for the treatment of synucleinopathies |
| WO2022076556A2 (en) | 2020-10-07 | 2022-04-14 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy disorders including limb girdle 2i (lgmd2i) |
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| CN117980326A (en) | 2021-07-14 | 2024-05-03 | 2赛文缇生物公司 | Engineered T cell receptors fused to binding domains from antibodies |
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| CA3234720A1 (en) | 2021-10-08 | 2023-04-13 | Sola Biosciences Llc | Compositions and methods for the treatment of p53-mediated cancers |
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| US6436392B1 (en) | 1998-05-20 | 2002-08-20 | University Of Iowa Research Foundation | Adeno-associated virus vectors |
| US6924365B1 (en) | 1998-09-29 | 2005-08-02 | Transkaryotic Therapies, Inc. | Optimized messenger RNA |
| AU782966B2 (en) * | 1999-06-08 | 2005-09-15 | University Of Iowa Research Foundation, The | Compounds and methods to enhance RAAV transduction |
| US7241447B1 (en) | 1999-10-07 | 2007-07-10 | University Of Iowa Research Foundation | Adeno-associated virus vectors and uses thereof |
| JP2006521825A (en) | 2003-03-31 | 2006-09-28 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | Compounds and methods for enhancing rAAV introduction |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| WO2007092944A2 (en) | 2006-02-08 | 2007-08-16 | Introgen Therapeutics, Inc. | Compositions and methods involving gene therapy and proteasome modulation |
| WO2007149852A2 (en) | 2006-06-19 | 2007-12-27 | Asklepios Biopharmaceutical, Inc. | Modified factor viii and factor ix genes and vectors for gene therapy |
| US7892824B2 (en) | 2007-01-18 | 2011-02-22 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma |
| WO2008127707A1 (en) * | 2007-04-13 | 2008-10-23 | Dana Farber Cancer Institute, Inc. | Receptor tyrosine kinase profiling |
-
2011
- 2011-02-04 JP JP2012552100A patent/JP6141021B2/en active Active
- 2011-02-04 CA CA2788682A patent/CA2788682C/en active Active
- 2011-02-04 US US13/575,975 patent/US9169492B2/en active Active
- 2011-02-04 WO PCT/US2011/023715 patent/WO2011097456A2/en active Application Filing
- 2011-02-04 EP EP11740407.9A patent/EP2531604B1/en active Active
- 2011-02-04 ES ES11740407.9T patent/ES2628889T3/en active Active
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